Search research articles
Contact Us
Filters
Showing results (11-20 of 57) with videos related to
Page
of 6
Sort By:
Clinical Genetics
|
March 28, 2025
Research-Based Whole Genome Sequencing Identifies Biallelic Loss of Function Variants in DOCK3 Gene Causing DOCK3-Related Disorder: The End of a Diagnostic Journey for This Family
Khurram Liaqat, Kayla Treat, Lili Mantcheva, et al.
Molecular Therapy : the Journal of the American Society of Gene Therapy
|
May 13, 2010
Vasoactive intestinal peptide increases hepatic transduction and reduces innate immune response following administration of helper-dependent Ad
Francesco Vetrini, Nicola Brunetti-Pierri, Donna J Palmer, et al.
Human Mutation
|
March 22, 2006
Aberrant splicing in the ocular albinism type 1 gene (OA1/GPR143) is corrected in vitro by morpholino antisense oligonucleotides
Francesco Vetrini, Roberta Tammaro, Sergio Bondanza, et al.
Molecular Therapy : the Journal of the American Society of Gene Therapy
|
January 30, 2013
SR-A and SREC-I are Kupffer and endothelial cell receptors for helper-dependent adenoviral vectors
Pasquale Piccolo, Francesco Vetrini, Pratibha Mithbaokar, et al.
American Journal of Medical Genetics. Part A
|
September 2, 2020
EVEN-PLUS syndrome: A case report with novel variants in HSPA9 and evidence of HSPA9 gene dysfunction
Georgianne Younger, Francesco Vetrini, David D Weaver, et al.
Clinical Genetics
|
November 14, 2020
Genotype-phenotype study and expansion of ARL6IP1-related complicated hereditary spastic paraplegia
Ye Cao, Melanie Manning, Kathleen Pope, et al.
Congenital Anomalies
|
March 23, 2024
Undiagnosed rare disease clinic identifies a novel UBE3A variant in two sisters with Angelman syndrome: The end of a diagnostic odyssey
Rebecca Bruns, Khurram Liaqat, Abdul Nasir, et al.
Human Gene Therapy
|
July 7, 2012
Sustained reduction of hyperbilirubinemia in Gunn rats after adeno-associated virus-mediated gene transfer of bilirubin UDP-glucuronosyltransferase isozyme 1A1 to skeletal muscle
Nunzia Pastore, Edoardo Nusco, Jana Vaníkova, et al.
Molecular Therapy : the Journal of the American Society of Gene Therapy
|
April 21, 2011
Generation of a Kupffer cell-evading adenovirus for systemic and liver-directed gene transfer
Reeti Khare, Shannon M May, Francesco Vetrini, et al.
Human Gene Therapy Methods
|
August 17, 2013
Improved efficacy and reduced toxicity by ultrasound-guided intrahepatic injections of helper-dependent adenoviral vector in Gunn rats
Nunzia Pastore, Edoardo Nusco, Pasquale Piccolo, et al.
Page
of 6
Search research articles
Search
Showing results (11-20 of 57) with videos related to
Sort By:
Page
of 6
Clinical Genetics
|
March 28, 2025
Research-Based Whole Genome Sequencing Identifies Biallelic Loss of Function Variants in DOCK3 Gene Causing DOCK3-Related Disorder: The End of a Diagnostic Journey for This Family
Khurram Liaqat, Kayla Treat, Lili Mantcheva, et al.
Molecular Therapy : the Journal of the American Society of Gene Therapy
|
May 13, 2010
Vasoactive intestinal peptide increases hepatic transduction and reduces innate immune response following administration of helper-dependent Ad
Francesco Vetrini, Nicola Brunetti-Pierri, Donna J Palmer, et al.
Human Mutation
|
March 22, 2006
Aberrant splicing in the ocular albinism type 1 gene (OA1/GPR143) is corrected in vitro by morpholino antisense oligonucleotides
Francesco Vetrini, Roberta Tammaro, Sergio Bondanza, et al.
Molecular Therapy : the Journal of the American Society of Gene Therapy
|
January 30, 2013
SR-A and SREC-I are Kupffer and endothelial cell receptors for helper-dependent adenoviral vectors
Pasquale Piccolo, Francesco Vetrini, Pratibha Mithbaokar, et al.
American Journal of Medical Genetics. Part A
|
September 2, 2020
EVEN-PLUS syndrome: A case report with novel variants in HSPA9 and evidence of HSPA9 gene dysfunction
Georgianne Younger, Francesco Vetrini, David D Weaver, et al.
Clinical Genetics
|
November 14, 2020
Genotype-phenotype study and expansion of ARL6IP1-related complicated hereditary spastic paraplegia
Ye Cao, Melanie Manning, Kathleen Pope, et al.
Congenital Anomalies
|
March 23, 2024
Undiagnosed rare disease clinic identifies a novel UBE3A variant in two sisters with Angelman syndrome: The end of a diagnostic odyssey
Rebecca Bruns, Khurram Liaqat, Abdul Nasir, et al.
Human Gene Therapy
|
July 7, 2012
Sustained reduction of hyperbilirubinemia in Gunn rats after adeno-associated virus-mediated gene transfer of bilirubin UDP-glucuronosyltransferase isozyme 1A1 to skeletal muscle
Nunzia Pastore, Edoardo Nusco, Jana Vaníkova, et al.
Molecular Therapy : the Journal of the American Society of Gene Therapy
|
April 21, 2011
Generation of a Kupffer cell-evading adenovirus for systemic and liver-directed gene transfer
Reeti Khare, Shannon M May, Francesco Vetrini, et al.
Human Gene Therapy Methods
|
August 17, 2013
Improved efficacy and reduced toxicity by ultrasound-guided intrahepatic injections of helper-dependent adenoviral vector in Gunn rats
Nunzia Pastore, Edoardo Nusco, Pasquale Piccolo, et al.
Page
of 6